[go: up one dir, main page]

EP3914261A4 - Rna-editing oligonucleotides and uses thereof - Google Patents

Rna-editing oligonucleotides and uses thereof Download PDF

Info

Publication number
EP3914261A4
EP3914261A4 EP20745855.5A EP20745855A EP3914261A4 EP 3914261 A4 EP3914261 A4 EP 3914261A4 EP 20745855 A EP20745855 A EP 20745855A EP 3914261 A4 EP3914261 A4 EP 3914261A4
Authority
EP
European Patent Office
Prior art keywords
rna
editing oligonucleotides
oligonucleotides
editing
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP20745855.5A
Other languages
German (de)
French (fr)
Other versions
EP3914261A1 (en
Inventor
Andrew W. Fraley
Steven ROBINETTE
Nessan Bermingham
Mallikarjuna Reddy Putta
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Korro Bio Inc
Original Assignee
Korro Bio Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Korro Bio Inc filed Critical Korro Bio Inc
Publication of EP3914261A1 publication Critical patent/EP3914261A1/en
Publication of EP3914261A4 publication Critical patent/EP3914261A4/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/554—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Botany (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP20745855.5A 2019-01-22 2020-01-22 Rna-editing oligonucleotides and uses thereof Pending EP3914261A4 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962795359P 2019-01-22 2019-01-22
US201962822527P 2019-03-22 2019-03-22
US201962900011P 2019-09-13 2019-09-13
PCT/US2020/014512 WO2020154344A1 (en) 2019-01-22 2020-01-22 Rna-editing oligonucleotides and uses thereof

Publications (2)

Publication Number Publication Date
EP3914261A1 EP3914261A1 (en) 2021-12-01
EP3914261A4 true EP3914261A4 (en) 2023-06-28

Family

ID=71736985

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20745855.5A Pending EP3914261A4 (en) 2019-01-22 2020-01-22 Rna-editing oligonucleotides and uses thereof

Country Status (9)

Country Link
US (2) US11479575B2 (en)
EP (1) EP3914261A4 (en)
JP (1) JP2022518731A (en)
KR (1) KR20210129645A (en)
CN (1) CN113573717A (en)
AU (1) AU2020211949A1 (en)
CA (1) CA3127243A1 (en)
TW (1) TW202042822A (en)
WO (1) WO2020154344A1 (en)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017211461B2 (en) * 2016-01-26 2023-01-12 Nissan Chemical Corporation Single-stranded oligonucleotide
CN109477103A (en) 2016-06-22 2019-03-15 ProQR治疗上市公司Ⅱ Single-stranded RNA-editing oligonucleotides
KR102501980B1 (en) 2016-09-01 2023-02-20 프로큐알 테라퓨틱스 Ⅱ 비.브이. Chemically Modified Single-Stranded RNA-Editing Oligonucleotides
CN118416088A (en) 2017-03-03 2024-08-02 加利福尼亚大学董事会 RNA targeting of mutations via inhibitory tRNAs and deaminases
CA3127241A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
CN113573717A (en) 2019-01-22 2021-10-29 科罗生物公司 Oligonucleotides for RNA editing and their uses
AU2020210900A1 (en) 2019-01-22 2021-08-12 Korro Bio, Inc. RNA-editing oligonucleotides and uses thereof
CA3159944A1 (en) 2019-12-02 2021-06-10 David HUSS Therapeutic editing
EP4150089A1 (en) * 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
CA3162416C (en) * 2020-05-15 2023-07-04 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4158018A1 (en) * 2020-05-26 2023-04-05 Shape Therapeutics Inc. Compositions and methods for genome editing
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024097674A2 (en) * 2022-11-02 2024-05-10 Aligos Therapeutics, Inc. Modified short interfering nucleic acid (sina) molecules and uses thereof
WO2024110565A1 (en) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
WO2024121373A1 (en) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
WO2024200472A1 (en) 2023-03-27 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024220934A1 (en) 2023-04-21 2024-10-24 University Of Rochester Nucleic acid molecules for pseudouridylation
WO2024256620A1 (en) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurodegenerative disease
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015172889A1 (en) * 2014-05-16 2015-11-19 Ugichem Gmbh Peptide nucleic acid monomers and oligomers
WO2017220751A1 (en) * 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
WO2018041973A1 (en) * 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5314893A (en) 1993-01-25 1994-05-24 Bristol-Myers Squibb Co. Antiviral tetrahydropyrans
EP1071753A2 (en) 1998-04-20 2001-01-31 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
WO2002020773A2 (en) 2000-09-06 2002-03-14 Mcgill University Chimeric antisense oligonucleotides of arabinofuranose analogues and deoxyribose nucleotides
US6878805B2 (en) 2002-08-16 2005-04-12 Isis Pharmaceuticals, Inc. Peptide-conjugated oligomeric compounds
US8192937B2 (en) 2004-04-07 2012-06-05 Exiqon A/S Methods for quantification of microRNAs and small interfering RNAs
US20110065774A1 (en) 2008-01-31 2011-03-17 Alnylam Pharmaceuticals Chemically modified oligonucleotides and uses thereof
AR090906A1 (en) 2012-05-02 2014-12-17 Merck Sharp & Dohme CONJUGATES CONTAINING TETRAGALNAC AND PROCEDURES FOR THE ADMINISTRATION OF OLIGONUCLEOTIDES
US9650627B1 (en) 2012-07-19 2017-05-16 University Of Puerto Rico Site-directed RNA editing
AU2015252895B2 (en) 2014-05-01 2021-07-15 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
WO2016094845A2 (en) 2014-12-12 2016-06-16 Woolf Tod M Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
WO2016097212A1 (en) 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
JP6624743B2 (en) 2015-07-14 2019-12-25 学校法人福岡大学 Site-specific RNA mutagenesis method, target editing guide RNA used therefor, and target RNA-target editing guide RNA complex
DE102015012522B3 (en) 2015-09-26 2016-06-02 Eberhard Karls Universität Tübingen Methods and substances for directed RNA editing
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3421467A4 (en) 2016-02-26 2019-08-07 Shionogi & Co., Ltd 5-phenylazaindole derivative having ampk activating effect
JP7069135B2 (en) 2016-06-15 2022-05-17 ユニヴァーシティー オブ ユタ リサーチ ファウンデーション Compositions and Methods for Using Albumin-Based Nanopharmaceuticals
GB2568182A (en) 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
WO2018134301A1 (en) 2017-01-19 2018-07-26 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in rna editing
JP2020511141A (en) 2017-03-15 2020-04-16 ザ・ブロード・インスティテュート・インコーポレイテッド Novel Cas13b ortholog CRISPR enzyme and system
CN110799645B (en) 2017-04-12 2024-08-02 博德研究所 Novel VI CRISPR ortholog and system
CN111417727A (en) 2017-05-18 2020-07-14 博德研究所 Systems, methods, and compositions for targeted nucleic acid editing
AU2018338318B2 (en) 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
WO2019111957A1 (en) 2017-12-06 2019-06-13 学校法人福岡大学 Oligonucleotides, manufacturing method for same, and target rna site-specific editing method
WO2019152416A1 (en) * 2018-01-30 2019-08-08 Metro International Biotech, Llc Nicotinamide riboside analogs, pharmaceutical compositions, and uses thereof
US20210079393A1 (en) 2018-02-14 2021-03-18 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for rna editing
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US20220073915A1 (en) 2018-06-29 2022-03-10 Eberhard-Karls-Universität-Tübingen Artificial nucleic acids for rna editing
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
KR102666695B1 (en) 2018-10-12 2024-05-23 페킹 유니버시티 Methods and compositions for editing RNA
WO2020092448A1 (en) 2018-10-29 2020-05-07 Spin Therapeutics, Llc Compositions and methods for alpha-1-antitrypsin disorders
CA3127241A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
CN113573717A (en) 2019-01-22 2021-10-29 科罗生物公司 Oligonucleotides for RNA editing and their uses
AU2020210900A1 (en) 2019-01-22 2021-08-12 Korro Bio, Inc. RNA-editing oligonucleotides and uses thereof
US20220112495A1 (en) 2019-01-28 2022-04-14 Proqr Therapeutics Ii B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
GB201901873D0 (en) 2019-02-11 2019-04-03 Proqr Therapeutics Ii Bv Antisense oligonucleotides for nucleic acid editing
EP4149518A4 (en) 2020-05-15 2024-08-21 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of abca4
KR20230033651A (en) 2020-05-28 2023-03-08 코로 바이오, 인크. Methods and compositions for ADAR-mediated editing of SERPINA1

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015172889A1 (en) * 2014-05-16 2015-11-19 Ugichem Gmbh Peptide nucleic acid monomers and oligomers
WO2017220751A1 (en) * 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
WO2018041973A1 (en) * 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
BAO T. LE ET AL: "Antisense oligonucleotide modified with serinol nucleic acid (SNA) induces exon skipping in mdx myotubes", RSC ADVANCES, vol. 7, no. 54, 2017, pages 34049 - 34052, XP055568101, DOI: 10.1039/C7RA06091B *
KASHIDA HIROMU ET AL: "Acyclic artificial nucleic acids with phosphodiester bonds exhibit unique functions", POLYMER JOURNAL, vol. 48, no. 7, 2016, pages 781 - 786, XP037325150, ISSN: 0032-3896, DOI: 10.1038/PJ.2016.39 *
KEIJI MURAYAMA ET AL: "Highly Stable Duplex Formation by Artificial Nucleic Acids Acyclic Threoninol Nucleic Acid ( a TNA) and Serinol Nucleic Acid (SNA) with Acyclic Scaffolds", CHEMISTRY - A EUROPEAN JOURNAL, vol. 19, no. 42, 11 October 2013 (2013-10-11), DE, pages 14151 - 14158, XP055568099, ISSN: 0947-6539, DOI: 10.1002/chem.201301578 *
PAUL VOGEL ET AL: "Improving Site-Directed RNA Editing In Vitro and in Cell Culture by Chemical Modification of the GuideRNA", ANGEWANDTE CHEMIE INTERNATIONAL EDITION, vol. 53, no. 24, 28 May 2014 (2014-05-28), pages 6267 - 6271, XP055763014, ISSN: 1433-7851, DOI: 10.1002/anie.201402634 *
See also references of WO2020154344A1 *

Also Published As

Publication number Publication date
US11479575B2 (en) 2022-10-25
US20200399303A1 (en) 2020-12-24
CA3127243A1 (en) 2020-07-30
AU2020211949A1 (en) 2021-08-12
CN113573717A (en) 2021-10-29
EP3914261A1 (en) 2021-12-01
JP2022518731A (en) 2022-03-16
US20230146891A1 (en) 2023-05-11
TW202042822A (en) 2020-12-01
KR20210129645A (en) 2021-10-28
US12152050B2 (en) 2024-11-26
WO2020154344A1 (en) 2020-07-30

Similar Documents

Publication Publication Date Title
EP3914260A4 (en) Rna-editing oligonucleotides and uses thereof
EP3914261A4 (en) Rna-editing oligonucleotides and uses thereof
EP3913001A4 (en) Anti-cld18a2 nanobody and application thereof
EP4041362A4 (en) Guidewire-management devices and methods thereof
EP3917497A4 (en) Oligonucleotide compositions and methods thereof
EP3917934A4 (en) Compounds and uses thereof
EP3918323A4 (en) Anti-gal3 antibodies and uses thereof
EP4076448A4 (en) Fluoroalkyl-oxadiazoles and uses thereof
EP3917517A4 (en) Compounds and uses thereof
EP3917529A4 (en) Compounds and uses thereof
EP3941908A4 (en) Compounds and uses thereof
EP4026846A4 (en) Anti-tigit immunosuppressant and application thereof
EP4034535A4 (en) Aza-quinoline compounds and uses thereof
EP3892621A4 (en) Haloallylamine compounds and application thereof
EP4032904A4 (en) Anti-alpha-hemolysin antibody and use thereof
EP4036107A4 (en) Mmup monomer variant and application thereof
EP3986935A4 (en) Anti-cd47 antibodies and uses thereof
EP4010368A4 (en) Anti-nampt antibodies and uses thereof
EP3914593A4 (en) Compounds and uses thereof
EP4036106A4 (en) Mnep monomer variant and application thereof
EP3794027A4 (en) Optimized gp41-binding molecules and uses thereof
EP3911322A4 (en) Compounds and uses thereof
EP3947365A4 (en) Substituted-n-heteroaryl compounds and uses thereof
IL284717A (en) Double stranded rna and uses thereof
EP4071171A4 (en) Anti-tm4sf4 antibody and use thereof

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20210804

AK Designated contracting states

Kind code of ref document: A1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40064153

Country of ref document: HK

REG Reference to a national code

Ref country code: DE

Ref legal event code: R079

Free format text: PREVIOUS MAIN CLASS: A61K0031712500

Ipc: C12N0015113000

RIC1 Information provided on ipc code assigned before grant

Ipc: C07H 21/00 19740701ALI20230111BHEP

Ipc: A61K 31/7088 20000101ALI20230111BHEP

Ipc: A61K 31/7125 20000101ALI20230111BHEP

Ipc: C12N 15/113 20100101AFI20230111BHEP

A4 Supplementary search report drawn up and despatched

Effective date: 20230601

RIC1 Information provided on ipc code assigned before grant

Ipc: C07H 21/00 20060101ALI20230525BHEP

Ipc: A61K 31/7088 20060101ALI20230525BHEP

Ipc: A61K 31/7125 20060101ALI20230525BHEP

Ipc: C12N 15/113 20100101AFI20230525BHEP